UX003

DRUG5 trials

Sponsors

Ultragenyx Pharmaceutical Inc, Joyce Fox

Conditions

MPS 7MPS VIIMucopolysaccharidosisMucopolysaccharidosis Type 7Mucopolysaccharidosis VIISly Syndrome

Phase 1

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

CompletedNCT01856218

Ultragenyx Pharmaceutical IncMucopolysaccharidosis Type 7

Start: 2013-11-30End: 2016-07-31Updated: 2019-01-04

Phase 2

Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age

CompletedNCT02418455

Ultragenyx Pharmaceutical IncMPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII +1

Start: 2015-07-21End: 2019-03-26Updated: 2019-10-30

Phase 3

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

CompletedNCT02230566

Ultragenyx Pharmaceutical IncMPS 7, MPS VII, Mucopolysaccharidosis +1

Start: 2014-12-31End: 2016-05-31Updated: 2020-07-30

A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)

CompletedNCT02432144

Ultragenyx Pharmaceutical IncMPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII +1

Start: 2015-11-10End: 2019-01-14Updated: 2020-07-30

Unknown Phase

An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient

Joyce FoxMucopolysaccharidosis Type 7

Updated: 2017-02-06

Related Papers

Growth patterns in patients with mucopolysaccharidosis VII.

2023-06-26

Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII.

2022-03-094 citations

The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases.

2021-04-1922 citations

The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII

Molecular Genetics and Metabolism2020-01-1135 citations

Pharmacokinetic and Pharmacodynamic Modeling to Optimize the Dose of Vestronidase Alfa, an Enzyme Replacement Therapy for Treatment of Patients with Mucopolysaccharidosis Type VII: Results from Three Trials.

2019-05-0117 citations